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Rosetta, Marina Biotech collaborate to identify & develop microRNA-based diagnostics and therapeutics for rare diseases

Princeton, New JerseySaturday, April 5, 2014, 14:00 Hrs  [IST]

Rosetta Genomics Ltd., a leading developer and provider of microRNA-based molecular diagnostics and therapeutics, and Marina Biotech, Inc., a leading nucleic acid-based drug discovery and development company, have established a strategic alliance, wherein the companies will collaborate to identify and develop microRNA-based products designed to diagnose and treat various neuromuscular diseases and dystrophies.

The Alliance is exclusive as it relates to neuromuscular diseases and dystrophies, with both companies free to develop and collaborate outside this field both during and after the terms of the Alliance. The companies' initial efforts are expected to be focused on Becker and Duchenne muscular dystrophies as well as myotonic dystrophy. Financial terms and other details of the agreement are not being disclosed at this time.

Under the terms of the Alliance, Rosetta will apply its industry leading microRNA discovery expertise for the identification of microRNAs involved in the various dystrophy diseases. If the microRNA is determined to be correlative to the disease, Rosetta may further develop the microRNA into a diagnostic for patient identification and stratification. If the microRNA is determined to be involved in the disease pathology and represents a potential therapeutic target, Marina may develop the resulting microRNA-based therapeutic for clinical development.

"We are delighted to have the opportunity to apply our leading-edge microRNA expertise with Marina's single- and double-stranded nucleic acid therapeutics in order to isolate specific microRNA biomarkers for the development of diagnostics and therapies for a variety of dystrophies. These rare diseases remain a significant unmet medical need," stated Kenneth A. Berlin, president and chief executive officer of Rosetta Genomics. "Targeted diagnostics to identify potential responders/non-responders for specific therapies would mark a significant advance for these patients as dystrophies are degenerative diseases that accelerate over time. In addition, current treatment options are sub-optimal and costly. We believe the development of a diagnostic that can accurately predict response to therapy will bring effective treatment to dystrophy patients earlier and with significant cost savings."

"The Alliance is a perfect match of cutting-edge technologies," stated J. Michael French, president and CEO of Marina Biotech. "Rosetta brings to the Alliance world class expertise in the development of microRNA diagnostics. Importantly, Rosetta can potentially identify novel microRNA targets for further development into clinical candidates. Marina is in the unique position to capitalize on Rosetta's expertise by utilizing Rosetta's microRNA-based diagnostics for patient identification and stratification in Marina's clinical development programs and by utilizing Marina's proprietary chemistries and delivery technologies to advance novel microRNA-based therapeutics -- both single-stranded microRNA antagonists and double-stranded microRNA mimics -- into the clinic. We believe our Smarticles is the only delivery technology currently in clinical development that is delivering both single-stranded oligonucleotides and double-stranded microRNA mimics. We believe this delivery technology, combined with our conformationally restricted nucleotide chemistry, will permit us to develop best-in-class microRNA antagonists and mimics. Our ability to work with both modalities is potentially critically important in the treatment of multi-system diseases such as myotonic dystrophy. While current technologies are limited by either a single-stranded or a double-stranded approach, Marina can pursue whichever nucleic acid modality most effectively treats these debilitating diseases."

"This latest strategic alliance underscores the versatility of Rosetta's microRNA biomarker platform for the identification of specific microRNA signatures for a variety of indications and applications and positions Rosetta Genomics as the partner of choice for such collaborations," added Berlin. "Importantly, these partnerships represent our company's third platform for revenue growth. Moving forward, we expect to enter into additional agreements as we seek to monetize our leading microRNA biomarker platform."

Neuromuscular disorders affect the nerves that control voluntary muscles, such as those that control the arms and legs. Nerve cells, also called neurons, send messages that control these muscles. When the neurons become unhealthy or die, communication between the nervous system and muscles breaks down. As a result, muscles weaken and waste away. Likewise, dystrophies are progressive degenerative disorders affecting skeletal muscles. In both cases, the diseases can often effect other organ systems such as the heart and central nervous system. Many neuromuscular diseases and almost all dystrophies are genetic, which means there is a mutation in genes which in many cases is passed from family member to family member. Although a cure for these disorders may present itself in the future, currently the goal of drug development efforts is to improve symptoms, increase mobility and increase the individual's lifespan.

Marina Biotech is an oligonucleotide therapeutics company with broad drug discovery technologies providing the ability to develop proprietary single and double-stranded nucleic acid therapeutics including siRNAs, microRNA mimics, antagomirs, and antisense compounds, including messengerRNA therapeutics.

Rosetta Cancer Tests are a series of microRNA-based diagnostic testing services offered by Rosetta Genomics. The Rosetta Cancer Origin Test? can accurately identify the primary tumor type in primary and metastatic cancer including cancer of unknown or uncertain primary (CUP). Rosetta Mesothelioma Test diagnoses mesothelioma, a cancer connected to asbestos exposure. The Rosetta Lung Cancer Test accurately identifies the four main subtypes of lung cancer using small amounts of tumor cells. The Rosetta Kidney Cancer Test accurately classifies the four most common kidney tumors: clear cell renal cell carcinoma (RCC), papillary RCC, chromophobe RCC and oncocytoma.

Founded in 2000, Rosetta's integrative research platform combining bioinformatics and state-of-the-art laboratory processes has led to the discovery of hundreds of biologically validated novel human microRNAs.

 
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